NCT04938635

Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia

A Phase 2b, Double-blind, Randomised, Placebo-controlled, Multicentre Study to Assess the Efficacy and Safety of VIT-2763 Multiple Doses in Adults With Transfusion-dependent Beta-thalassaemia

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Vifor (International) Inc. · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The main purpose of this study is to evaluate the efficacy of 3 multiple doses of VIT-2763 as measured by the reduction in red blood cell (RBC) transfusion burden from Week 13 to Week 24, to identify the most efficacious and safe dose.

Detailed description

All patients giving written informed consent will undergo a 12-week screening period to determine eligibility for study entry. At Day 1, patients who meet the eligibility requirements will be randomized in a double-blind manner to 1 of 4 treatment groups: VIT-2763 60 mg (once daily), 60 mg (twice daily), or 120 mg (twice daily) or placebo. The randomisation will be stratified (balanced allocation across treatment groups) according to β0/β0 genotype (yes/no). The duration of the treatment with VIT-2763 or placebo is 24 weeks, after which patients will undergo a 12-week post-treatment follow-up period.

Conditions

• Beta-Thalassemia

Interventions

Type	Name	Description
DRUG	VIT-2763 60 mg QD	Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally in the morning, and 2 Placebo capsules orally in the evening, for 24 weeks.
DRUG	VIT-2763 60 mg BID	Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally, twice daily for 24 weeks.
DRUG	VIT-2763 120 mg BID	Participants receive two VIT-2763 60 mg capsules orally, twice daily for 24 weeks.
DRUG	Placebo	Participants receive two Placebo capsules matching VIT-2763 orally, twice daily for 24 weeks.

Timeline

• Start date: 2021-09-20
• Primary completion: 2023-07-01
• Completion: 2023-07-01
• First posted: 2021-06-24
• Last updated: 2022-11-10

Locations

7 sites across 3 countries: United States, Bulgaria, Israel

Regulatory

• FDA-regulated drug study